National Center for Gene Therapy and Drugs based on RNA Technology


Pierpaolo Ceci



Istituto di Biologia e Patologia Molecolari



Objectives of the project -Long noncoding RNAs (lncRNAs) are expressed in a highly specific manner in cancer, where they function as oncogenes or oncosuppressors. This makes them powerful candidates as potential therapeutic targets.  Human ferritin (HFt) is a multimeric protein-cage that self-assembles into a structure able to encapsulate drugs and to deliver them to CD71(TfR1)-expressing cancer cells. Recently, the successful encapsulation of siRNAs into HFt was reported, the HFt-siRNAs complexes being able to efficiently silence target genes both in vitro and in vivo. We will exploit HFt-based platform to bind and selectively deliver siRNA or AntiSense Oligonucleotides (ASO) molecules to the tumor target cells.

We will apply a combination of:

  • computational predictions for the design of therapeutic small RNAs
  • cell manipulation by reverse genetics (loss-of-function) approaches
  • biochemical/biophysical techniques to produce and characterize siRNA-ferritin complexes
  • integrated molecular (gene expression) and phenotypic (cell viability/death) assays in cancer cells to test small RNA efficacy


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